Alnylam Pharmaceuticals and the Ludwig Maximilian University Munich Publish Data on Short Interfering RNAs and Immune Stimulation Mechanisms
Tuesday February 22, 8:02 am ET
Scientific Insights Published in Nature Medicine Advance the Development of RNAi Therapeutics
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 22, 2005-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY - News), a leading RNAi therapeutics company, and its collaborators at the Ludwig Maximilian University Munich announced today the online publication of an article in Nature Medicine identifying specific immunostimulatory motifs of short interfering RNAs (siRNAs), the molecules that induce RNA interference. The identification of these motifs enables the design of siRNAs that avoid interferon induction, an important goal for achieving safe RNAi therapeutics. These data have applications for deciphering the biological mechanisms of gene silencing and understanding how to design and optimize siRNA sequences that are bothsafe and efficacious as therapeutics.
In the paper, immune stimulation, as measured by interferon induction, was found to occur in association with some siRNA sequences but not others. These findings were observed in vivo in association with the use of liposomal delivery of the siRNAs. In connection with the Nature Medicine publication, Alnylam has licensed certain intellectual property relating to Dr. Gunther Hartmann's work from the Ludwig Maximilian University Munich.
"Understanding the sequence-specific regulation of the immune response toward short interfering RNA is a tremendous advancement in RNAi biology and the development of therapeutics based on RNAi," said Dr. Hartmann, M.D., Division of Clinical Pharmacology, Ludwig Maximilian University Munich Hospital, Germany.
"Alnylam is committed to developing safe and efficacious RNAi therapeutics," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam Pharmaceuticals. "Elucidating the mechanism of action responsible for siRNA immunostimulation will allow for efficient selection of appropriate siRNA molecules."
About RNA Interference (RNAi)
RNA interference, or RNAi, is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence and regulate such genes selectively through RNAi could provide a means to treat a wide range of human diseases. The discovery of RNAi has been heralded by many as a major breakthrough, and the journal Science named RNAi the top scientific achievement of 2002, as well as one of the top 10 scientific advances of 2003.
About Alnylam
Alnylam is a biopharmaceutical company seeking to develop and commercialize novel therapeutics based on RNA interference, or RNAi. Growing from its foundation as the world's first company focused on RNAi therapeutics, the company's leadership in the field of RNAi is supported by its preeminent founders and advisors and its strengths in fundamental patents, technology, and know-how that underlie the commercialization of RNAi therapeutics. Alnylam is developing a pipeline of RNAi products using Direct RNAi(TM) to treat ocular, central nervous system, and respiratory diseases and Systemic RNAi(TM) to treat a broad range of diseases, including oncology, metabolic, and autoimmune diseases. The company's global headquarters are in Cambridge, Massachusetts. For additional information, please visit www.alnylam.com. |
